Myelodysplastic Syndrome Clinical Trial
BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy
Summary
The purpose of this study is to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH patients with residual anemia despite treatment with a C5 inhibitor.
Full Description
This is a randomized, active comparator-controlled, open-label, parallel-group, 2-part study. Parts 1 and 2 will be conducted in the same subjects.
Part 1 of the study is designed to evaluate the efficacy, safety, and tolerability of oral BCX9930 monotherapy for 24 weeks versus continuing C5 inhibitor therapy in subjects with PNH with inadequate response to their current C5 inhibitor therapy. Subjects will be randomized to either discontinue C5 inhibitor therapy and start BCX9930 monotherapy or to continue C5 inhibitor therapy for the 24-week randomized treatment period. The primary efficacy and safety analyses will be based on Part 1.
Part 2 of the study is designed to evaluate the long-term safety, tolerability, and effectiveness of BCX9930 monotherapy when administered through Week 52. All subjects in Part 2 will receive BCX9930. Subjects who are randomized to BCX9930 monotherapy in Part 1 will continue to receive BCX9930 in Part 2. Subjects who are randomized to C5 inhibitor therapy in Part 1 will discontinue that therapy at the Week 24 visit and receive BCX9930 in Part 2.
Eligibility Criteria
Inclusion Criteria:
Male or female, aged ≥ 18 years old
Body weight ≥ 40 kg
Documented diagnosis of PNH
Currently being treated with a stable C5 inhibitor regimen
Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willing to start vaccination series
At screening: PNH clone size of ≥ 10% and hemoglobin ≤ 10.5 g/dL
Exclusion Criteria:
Known history of or existing diagnosis of hereditary complement deficiency
History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation
Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition
History of malignancy within 5 years prior to the screening visit
Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening
Treatment with anti-thymocyte globulin within 180 days prior to screening
Initiation of treatment with an erythrocyte or platelet growth factor, or danazol within 28 days prior to screening
Receiving iron supplementation with an unstable dose in the 28 days prior to screening
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There is 1 Location for this study
Paris , , France
Budapest , , Hungary
Rome , , Italy
Barcelona , , Spain
Valencia , , Spain
Leeds , , United Kingdom
London , , United Kingdom
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